Much of the attention in the genetic editing world in recent years has been on CRISPR and the “genetic scissors” that allow researchers to make targeted changes to DNA. In 2020, scientists Jennifer Doudna and Emmanuelle Charpentier won the Nobel Prize in Chemistry for their work on CRISPR, and a raft of biotech companies focused on the technology have advanced into clinical testing.
Initial results have been encouraging. Earlier this summer, a CRISPR medicine developed by Intellia Therapeutics and Regeneron Pharmaceuticals provided the first clinical evidence that the gene editing technology could work when delivered systemically into the body. Data from CRISPR Therapeutics and Vertex, meanwhile, has shown great potential to using CRISPR on cells extracted from the body and then reinfused to treat the blood diseases sickle cell and beta-thalassemia.
Lilly, which previously was not invested heavily in the field, has begun to take an interest in DNA editing as well, licensing technology from Precision Biosciences last year.
But along with the optimism comes concerns about the unknowns of making permanent changes in DNA. Proponents of RNA editing claim it offers a way to address genetic mutations with less risk because it’s temporary and reversible.
The promise of RNA editing has sparked multiple startups, including Shape Therapeutics and Korro Bio. Shape already snagged a partnership with Roche that may be worth as more than $3 billion if the companies are successful in developing treatments for diseases including Alzheimer’s and Parkinson’s.
Both ProQR and Lilly have been working on RNA therapies for some time. In January, ProQR announced it had completed enrollment of a pivotal trial for its sepofarsen treatment for a rare genetic condition that leads to blindness. The company’s eye injection works by binding to mutated RNA to allow splicing needed to produce an essential protein for vision.
Lilly, meanwhile, has signed a number of deals focused on RNA-based medicines in recent years. The company in May announced an agreement with MiNA Therapeutics to collaborate on as many as five targets using that company’s small activating RNA technology.
